Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

The present study examines the safety profile of intravenous onasemnogene abeparvovec gene therapy in a real-world setting, both alone or in combination with intrathecal antisense oligonucleotide ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disorder marked by hypotonia, progressive muscle weakening, and areflexia. This condition stems from a homozygous deletion of the ...